Barry Schatz thought losing weight was a sign his exercise plan was working. Instead, the 47-year-old father of two learned in 2000 that he was losing weight due to chronic myelogenous leukemia, a cancer of the blood-forming cells of the bone marrow.
“My diagnosis came completely out of left field,” says Schatz, Associate Director for Administration at Loyola University Chicago’s Cardinal Bernardin Cancer Center. “When I showed my white blood cell count to my boss, a cancer physician, he thought that I had mistakenly written down too many zeros.”
Schatz was fortunate to get treatment through a clinical trial that gave him Gleevec, which has since become a first-line therapy. However, after about a year, it stopped working, and a bone marrow transplant became Schatz’s only option for survival. He received the transplant, in which healthy donor marrow replaces a patient’s diseased marrow, at the cancer center where he works. It was a success.
The process made Schatz realize he wanted to do even more than he already was doing for people with cancer in his position at Loyola. So eventually, he became a partner on a PCORI-funded project working to identify and build the foundation for studying the impact of transplants on recipients’ quality of life.
Until recently, patients, clinicians, and researchers considered a bone marrow transplant successful if the recipient lived. Fortunately, because the number of transplant survivors has increased, the research spotlight is now also focusing on the near- and long-term challenges survivors face.
After his diagnosis, Schatz’s concerns turned toward his family—his wife and children, who were then 10 and 7. That focus has extended to the work he’s doing with support from a PCORI Engagement Award to the National Marrow Donor Program. The project looks to “add the views of patients and their families to the clinical perspectives that medical people generally bring to research,” he says.
A Growing Community, a Changing Focus
Bone marrow transplantation is generally not first-line treatment for marrow and other blood cancers. It can require weeks to months of hospitalization for monitoring and treatment and it can have many side effects, including pain, fever, chills, nausea, bleeding, and infection risk. Some can be life-threatening.
Instead of saying, ‘Here’s what we think researchers and patients need,’ we said, ‘Let’s ask the patients.'Linda Burns, MD Project lead, PCORI-funded engagement project
But more and more transplant recipients are surviving. In the past five years, the number of patients alive one year after receiving a transplant from an unrelated donor increased from 42 to 60 percent, according to the National Marrow Donor Program. In 2009, there were approximately 110,000 transplant survivors; experts expect around 240,000 survivors by 2020.
Linda Burns, MD, Vice President and Medical Director of Health Services Research at the National Marrow Donor Program, has watched the transplant world’s focus shift from curing people of a serious disease to also recognizing and addressing long-term effects of those cures.
“In the 1990s, I cared for a young patient who developed severe osteoporosis several years after receiving a bone marrow transplant. The patient broke many ribs from just rolling over in bed,” says Burns, a medical oncologist and hematologist who performed her first transplant in 1984. “This was before we understood that survivors of transplant can have weakened bones.”
Burns’s experience made her want to know more about improving the quality of patients’ lives. “Instead of saying, ‘Here’s what we think researchers and patients need,’ we said, ‘Let’s ask the patients,’” she says.
Identifying Research Questions
To try to do that, Burns and her team held three symposia with patients, caregivers, clinicians, researchers, and other stakeholders to find out what questions about bone marrow transplant outcomes they thought should be top research priorities.
Participants divided into work groups that identified knowledge gaps, took part in webinars about patient-centered outcomes research, and discussed potential research questions.
The work groups developed research questions on six topics: physical health, including fatigue; emotional, cognitive, and social health; sexual health and relationships; care delivery; financial burden; and education and support for patients, caregivers, and families.
Learning about Life beyond the Procedure
Through a PCORI-funded Engagement Award, patients, caregivers, and other stakeholders involved in the bone marrow transplant process produced a research agenda. Their high-priority research questions include the following:
- What is the most effective delivery method for education about the emotional and physical aspects of recovery from bone marrow transplant?
- Does the distance from patients’ and caregivers’ homes to a bone marrow transplant center predict the strength of their belief in themselves to handle the transplant process?
- What are the potential benefits of a peer support group for adolescents and young adults facing chronic illnesses and receiving bone marrow transplants?
- What interventions can prepare survivors for communicating their health history to current and future romantic partners?
Speaking from Experience
Schatz joined the family-centered work group. He says sometimes families don’t receive the support they need to address ongoing emotional and health issues, such as the skin rashes and immunity concerns he still experiences.
“The family is impacted tremendously,” he says. “Not only was my wife impacted on an emotional basis with considering the illness and potential death of her spouse, but she also needed to become the primary—and at times, sole—emotional support for our young children. She also had to take on my family responsibilities, as well as coordinate caregivers for me while I recovered.”
The work group zeroed in on families’ needs for emotional support and educational material. The group suggested family members receive tools for dealing with stress, for example. Participants thought an important research question would address which methods best provide such support, such as whether one-on-one or group settings were more effective.
Another participant in the work group, Susan Kullberg, a retired teacher, says hospital support groups sound helpful but can sometimes cause stress. Hearing about others’ experiences, especially in cases of transplant failure or death, can weigh heavily on families, she says.
When Kullberg was a teenager, her father died of Hodgkin lymphoma, another blood cancer. Decades later, she cared for her young grandchildren and 34-year-old daughter, Betsy, when Betsy underwent a transplant for chronic myelogenous leukemia.
“In the mid-1960s when my father was diagnosed, no one talked about cancer,” Kullberg says. “When Betsy was diagnosed in 2005, I thought there would be some kind of emotional support for our family. Actually, there was so little.” It would have helped her stay hopeful, she says, to have met someone who had survived chronic myelogenous leukemia. She now volunteers to provide just that kind of support for patients and families.
The work group also talked about the importance of educational materials, suggesting they take into account differences in cultures and consider different formats, such as videos versus traditional print. Ultimately, the group posed several research questions for further study, including how levels of distress compare for transplant patients and their families who receive provider-directed education versus those who receive both provider- and self-directed education.
An Agenda for Action
The participants in the PCORI-funded project want to see their work have a long reach. After each symposium, the team sent updates about the project to members of the American Society for Blood and Marrow Transplantation, the Center for International Blood and Marrow Transplant Research, the National Marrow Donor Program, and various transplant centers.
The symposia’s educational webinars are available for public use on the National Marrow Donor Program’s website. The team recently published its research recommendations in the journal Biology of Blood and Marrow Transplantation. Each work group also aims to develop at least one of their questions into a research proposal.
Contributing to a project like this one makes Schatz feel that, 17 years after his diagnosis, more of the healthcare community is joining him in recognizing that there is more to bone marrow transplantation than survival. “The first question when you are diagnosed is, ‘Am I going to live?’” he says. “Now, we also ask, ‘How well am I going to live?’”
At a Glance
Engaging Patients in Developing a Patient-Centered Hematopoietic Cell Transplant Research Agenda
Project Leads: Linda Burns, MD
Goal: To develop a research agenda for studying the bone marrow transplant outcomes that are most important to patients and their families.
Posted: March 20, 2018
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